In a paper publish in December 2023, we demonstrate a first step towards the development of a targeted, light-mediated gene therapy for the eyes: we show here that in vivo photoactivated transgene expression in retinas is now possible. Indeed, based on the inducible Cre/lox system, we introduced EE2 (approved for use in humans) as a substitute for Tamoxifen. EE2 was modified with a caging moiety that can be photolyzed at 450 nm and that way we can target viral transgene expression specifically to the pathological regions of the retina with light.
This scientific result has been published in Angewandte Chemie International Edition. Towards a light-mediated gene therapy for the eye - retinal transgene expression through photoactivation of caged ethinylestradiol and the inducible Cre/lox system, Z. Kiy, J. Chaud, L. Xu, E. Brandhorst, T. Kamali, H. Hong, A. Specht, S. Cambridge, Angew Chem Int Ed,2024, e202317675.
